This article discusses the scope and purpose of the special designation for Regenerative Medicine Advanced Therapies (RMAT) created by the passage of the 21st Century Cures Act. The authors explain the benefits expected to be realized with RMAT, such as keeping the US globally competitive in the field. They provide a tally of products receiving the special designation to date and a current count, by year, of products for which RMAT designation has been requested.

Introduction

Section 3033 of the 21st Century Cures Act, titled “Accelerated Approval for Regenerative Advanced Therapies,” created a special designation for Regenerative Medicine Advanced Therapies (RMAT). A product is eligible for RMAT designation if it is a regenerative medicine therapy, such as cell therapy, therapeutic tissue engineering product, human cell and tissue product, gene therapy or any combination product using such therapies or products, and is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.

Other national bodies have in recent years made special regulatory provisions to recognize the value of regenerative medicine products. For example, Japan’s SAKIGAKE designation, was introduced in 2015 to promote R&D of regenerative medicines and other innovate pharmaceuticals and medical devices.

RMAT designation has helped keep the US competitive in the global field, while addressing specific needs and requirements of regenerative medicine advanced therapy products. The benefits of an RMAT designation are the same as the benefits for breakthrough therapies and include interactions with FDA to expedite development and review of the product and consideration of the product for priority review or accelerated approval. Receiving RMAT designation also allows for increased flexibility in clinical trial design, for instance, in the number of clinical trial sites. Developers also have the potential to use patient registry data and other real-world sources in post-approval pathways.

While developers seeking breakthrough designation are required to show that their therapeutic candidate would provide a substantial improvement over existing therapies, RMAT designation only requires that the therapy have the potential to address unmet medical need.

With RMAT designations available for more than two years, what have we seen so far?

Table 1 provides FDA’s latest tally of RMAT designations by year. Only 2018 represents a full year. For 2019, the number of requests is at a higher rate (19 requests through 10 April 2019) but the number granted is about the same rate as for 2018 (about 1.5 requests granted per month).

Table 1. Metrics on RMAT Requests by Year1

Fiscal Year

Total Requests
Received

Granted

Denied

Withdrawn

2017

31

11

18

2

2018

47

18

27

2

2019

19

4

8

1

So far, no RMAT-designated products have received marketing approval and no RMAT designations have been reported withdrawn or rescinded.

Figure 1 provides metrics on designation requests by therapeutic area as of September 2018. The largest category is neurology, followed by oncology. This is surprising because scientific publications of regenerative medicines for oncology far outpace those for neurology.2

Figure 2 provides metrics on designation requests by product type as of March 2019. The greatest number of designation requests have been for cell therapy products with allogeneic products outpacing autologous products.

Figure 1. RMAT Designation Requests by Therapeutic Area3

Figure 2. RMAT designation requests by product type4

Table 2 provides a listing of the publicly announced RMAT designations. Twenty-eight have been announced so far. There have been 33 RMAT designations granted as of 1 April 2019,3 thus five are currently unannounced.

In the following month, the Alliance for Regenerative Medicine plans to launch a database utilizing publicly available and company-provided information to create a public list of RMAT recipients, as well as other expedited approval designations awarded in the United States, Europe, and Japan.

Clearly, the RMAT designation program has been very active for FDA and popular for sponsors. We look forward to seeing the first products approved under this program.

From Wilson Bryan presentation to the Alliance for Regenerative Medicine Liaison Meeting on 28 March 2019.

Janet Lynch Lambert joined the Alliance for Regenerative Medicine (ARM) in 2017 as the organization’s first CEO. She most recently served as the acting head of engagement for the All of Us Research Program at the National Institutes of Health and as head of the Outreach Office in the Office of the NIH Director. Prior to joining NIH, she was vice president of government relations and head of the Washington office of Life Technologies.

William Sietsema is vice president, global regulatory affairs at Caladrius Biosciences, a company that focuses on innovative cell therapies for difficult-to-treat diseases. Prior to Caladrius, he was global regulatory lead at Amgen and vice president, global regulatory consulting and submissions at Kendle International/INC Research and adjunct professor of pharmaceutical sciences at the University of Cincinnati, College of Pharmacy. He may be contacted at william@sietsema.com.

First published in Regulatory Focus™ by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products.Click here for more information.

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